Table 1.
Comparison of clinical data
| Clinical data | Twin #1 | Twin #2 |
|---|---|---|
| Disease progression | ||
| Age of presentation | 2 | 2 |
| Age at transplantation (years) | 9 | 11 |
| Sex | Female | Female |
| Diagnosis | Constitutional SAA with trisomy 8 | Constitutional SAA with trisomy 8 |
| Karyotype at transplant | Trisomy 8 | Trisomy 8 |
| Pre-transplantation characteristics | ||
| Absolute neutrophil count (k/μl) | 417 | 996 |
| Absolute reticulocyte count(k/μl) | 61 | 71.2 |
| Platelets (k/μl) | 16 | 15 |
| Time from diagnose to PBSC (years) | 7 years | 9 years |
| Recipient blood type | AB+ | AB+ |
| Donor relationship | Brother | Brother |
| Donor blood type | A+ | A+ |
| Donor sex | Male | Male |
| HLA match | 9 out of 10 | 9 out of 10 |
| RIC allogeneic PBSC | ||
| Age at transplant | 9 | 11 |
| Recipient blood type | AB+ | AB+ |
| CD34+ cell dose (×106/kg) | 6.24 × 10.6 | 6.32 × 10.6 |
| CD3+ cell dose (×107/kg) | 42.3 × 10.7 | 35.2 × 10.7 |
| Day of 100 % donor T cell chimerism | Day +352 | Day +30 |
| Received DLI and stem cell boost | Yes | No |
| Prophylaxis against GVHD | MTX 5 mg/m2 day +1, +3, +6 | MTX 5 mg/m2 day +1,+3, +6 |
| Cyclosporine | Cyclosporine | |
| Conditioning regimen comparison | ||
| Cyclophosphamide 60 mg/kg IV × 2 | Yes | Yes |
| Fludarabine 25 mg/m2 IVBP × 5 | Yes | Yes |
| Rabbit ATG 3.5 mg/kg IV × 4 | Yes | No |
| Horse ATG 40 mg/kg IV × 4 | No | Yes |
| GVHD prophylaxis | ||
| MTX 5 mg/m2 × 3 | Yes | Yes |
| Cyclosporine | Yes | Yes |
| Post-transplantation GVHD | ||
| Time from HSCT to GVHD (months) | 20 months | <1 month |
| Acute GVHD | No | Yes (grade II GI tract) |
| Site of cGVHD | Skin, mouth | Skin, ocular, and lung |
| cGVHD grade (severity) | Limited | Extensive |
| cGVHD treatment | Daclizumab, cyclosporine | Cyclosporine, sirolimus, MMF, steroid |
| Daclizumab, rituximab, tacrolimus | ||